Kornimate/medical-research-clean huggingface dataset:
mt2015-20: biochemical correction of severe eb by allo hsct and serial donor mscs

Conditions: Epidermolysis Bullosa

Summary: this is a single-institution, phase ii study to determine the event-free survival at 1 year post allogeneic transplant and serial mesenchymal stem cell (msc) infusions from a related donor (hla identical, mismatched or haploidentical) or matched unrelated donor for the biochemical correction of severe epidermolysis bullosa (eb).


Eligibility criteria:
inclusion criteria: * diagnosis of severe form of eb characterized by collagen, laminin, integrin, keratin or plakin deficiency (by immunofluorescence staining with protein specific antibodies or western blotting and by mutation analysis). * adequate organ function within 4 weeks of study registration defined as: * renal: glomerular filtration rate within normal range for age * hepatic: hepatic: bilirubin, ast/alt, alp \< 5 x upper limit of normal * pulmonary: adequate pulmonary function in the opinion of the enrolling investigator * cardiac: left ventricular ejection fraction ≥ 45%, normal ekg or approved by cardiology for transplant * sexually active participants must agree to use adequate birth control for the during the study period (from before the start of the preparative chemotherapy through 1 year post-transplant) * available donor per section 5: targeted mfi \< 1,000 (mfi exceeding 1000 must be approved by the pi and treatment team.) * voluntary written consent - adult or parent (with information sheet for minors, if applicable) prior to any research related procedures or treatment exclusion criteria: * beta 3 laminin jeb mutants * active untreated systemic infection at time of transplantation (including active infection with aspergillus or other mold within 30 days) * history of hiv infection * evidence of squamous cell carcinoma * pregnant or breast feeding. females of child-bearing potential must have a negative pregnancy test prior to study registration as the agents administered in this study are pregnancy category c and d.
prone positioning for invasively ventilated patients with covid-19 registry

Conditions: COVID-19 Pneumonia

Summary: sars-cov-2 infection is characterized mainly by moderate/severe pneumonia associated with progressive endothelial damage and coagulopathy. acute respiratory failure among covid-19 occurs in 42% of patients with covid-19 pneumonia, and 61-81% of patients requiring intensive care . among the suggested treatments for the management of ards patients, prone position (pp) can be used as an adjuvant therapy for improving ventilation in these patients, as recommended in the surviving sepsis campaign covid-19 guidelines. nevertheless, no data is currently available on application and feasibility of pp in invasively ventilated patients with covid-19 on their outcomes.


Eligibility criteria:
inclusion criteria: * laboratory-confirmed covid-19 infection * prone positioning in patients admitted to icu undergoing invasive mechanical ventilation exclusion criteria: * bedsides contraindications to the prone positioning and patients in pp but undergoing non- invasive ventilation should be excluded.
treatment of endometrial hyperplasia with an intrauterine device (iud)

Conditions: Endometrial Hyperplasia

Summary: the purpose of this research is to determine the effectiveness of the intrauterine device (iud) called mirena when compared to the provera tablets used in treating this condition.

endometrial hyperplasia without atypia is a condition usually caused by excessive stimulation of the uterine lining (endometrium) by estrogens, and may cause irregular bleeding as a presenting symptom, but rarely may progress to endometrial cancer, which makes treatment important. the only treatment approved so far is medroxyprogesterone acetate tablets (progesterone), also known as provera.

Eligibility criteria:
inclusion criteria: * histologic diagnosis of simple or complex endometrial hyperplasia without atypia * normal pap smear within one year exclusion criteria: * diabetes * family history of endometrial cancer * contraindications for the intrauterine device

Keywords: endometrial hyperplasia without atypia
outcomes of stem cells for cerebral palsy

Conditions: Cerebral Palsy

Summary: the aim of this study is to assess the safety and effectiveness of autologous bone marrow mononuclear stem cells in patients with cerebral palsy.

the aim of this study is to assess the safety and effectiveness of autologous bone marrow mononuclear stem cells in 40 patients with cerebral palsy at vinmec international hospital, hanoi, vietnam.

Eligibility criteria:
inclusion criteria: * cerebral palsy of any types caused by oxygen deprivation. exclusion criteria: * epilepsy * hydrocephalus with ventricular drain * coagulation disorders * allergy to anesthetic agents * severe health conditions such as cancer, failure of heart, lung, liver or kidney * active infections

Keywords: cerebral palsy stem cells
genes modulating the severity of aortic aneurysms (msf1-tgfbr2)

Conditions: Thoracic Aortic Aneurysm

Summary: this project concerns a population at risk of sudden death by dissection of the thoracic aorta. its interest is to make it possible to recognize the genes that protect or worsen the evolution of aneurysms, to better understand the mechanisms involved, to detect and treat aneurysms of the thoracic aorta, wich is a pathology that is completely silent clinically until life-threatening complications. the variability in the severity of the disease within the same family is related to modifier genes. the objective is to find the modifying factors that account for the variability in the severity of the progression of aneurysms of the thoracic aorta.

thoracic aortic aneurysms are silent, asymptomatic, potentially fatal pathologies due to the risk of aortic dissection. more and more often they are found during imaging tests done for another reason. some aneurysms have genetic origin (autosomal dominantly inherited) and are particularly interesting because they can be recognized early (due to possible family screening), which allows us to understand the natural history of this pathology. the discovery of genes whose mutations explain the occurrence of these family aneurysms (initiator gene) has also made it possible to improve family screening and to better understand the pathophysiology of these aneurysms: we now recognize 3 groups of genes involved (extracellular matrix, contractile proteins of smooth muscle cell, tgf-β pathway (transforming growth factor) \[including mutations in tgf-β receptor 2 gene, tgfbr2\]). the variability in the severity of signs and aortic involvement is particularly marked in patients with aortic aneurysms due to mutations in the tgfbr2 gene. some patients with these mutations present aggressive aneurysms with early dissection. other patients have isolated late-onset aneurysms, and others have no signs. this variability generates problems for clinical practice to give appropriate genetic advice, but also to adapt imaging monitoring, therapy, or sports restriction. the present protocol aims is to investigate the variability in the severity of the disease within a large family carrying a mutation in the tgfbr2 gene. the mfs1 family is a family in which the aortic pathology is due to a mutation in the tgfbr2 gene. all patients with this family carry the same tgfbr2 mutation, heterozygous.

Eligibility criteria:
inclusion criteria: member of msf1 family. the mfs1 family is a family in which the aortic pathology is due to a mutation in the tgfbr2 gene. all patients with this family carry the same tgfbr2 mutation (heterozygous) exclusion criteria: refusal or linguistic or psychological inability to sign informed consent

Keywords: vascular diseases, aneurysm tgfbr2 mutation
coping skills training (cst) for children with chronic health conditions

Conditions: Rheumatologic Conditions (JRA,Lupus); Epilepsy; Spina Bifida; Asthma

Summary: purpose of the study the purpose of this study is to pilot an adapted coping skills training (cst) intervention for feasibility and preliminary efficacy with a sample of children 8 to 12 years of age and their parents. the participants in this study at children's hospital of wisconsin are dealing with one of three chronic health conditions (rheumatologic conditions, epilepsy,spina bifida, and asthma). research questions/study aims the research questions addressed in the full study are: 1. what is the impact of cst on child depression, qol, health motivation, attitude toward illness, and self-management efficacy? 2. what is the impact of cst on parent depression, perception of child's quality of life, perception of impact of chc on family, and family conflict?

many children with chronic health conditions (chc) are at increased risk for poor adaptation such as psychosocial problems, behavioral disturbances, and decreased quality of life (qol). their parents face economic, social and emotional challenges. in addition, management of the chc and the involvement of the child in that management can severely challenge both child and parent. effective coping has been shown to moderate the negative impact of chc. this study is a pilot study to evaluate the feasibility and preliminary efficacy of adapting a coping skills training (cst) intervention developed for children with diabetes. the cst intervention will be adapted for an integrated sample of school-aged children 8 to 12 years of age with four health conditions (rheumatologic conditions, epilepsy,spina bifida, and asthma). the study will be a randomized clinical trial with a wait-list control group. each arm will consist of 25 families. cst is a 6-session group intervention based on cognitive behavioral and learning theory. the impact of cst on both outcomes (child: depression, qol; parent: depression, child qol, chc impact on family) and protective factors (child: health motivation, attitude, self-management efficacy; parent: family conflict) will be measured.

Eligibility criteria:
inclusion criteria: * male and female children ages 8 to 12 with no known severe cognitive delays, * who are english speaking, * with one of the three target conditions; and * have at least one parent willing to participate. exclusion criteria: * children with cognitive delay

Keywords: coping psychosocial quality of life problem solving
safety study of lbh589 when given in combination with lenalidomide and dexamethasone in adult patients with multiple myeloma.

Conditions: Multiple Myeloma

Summary: this study will evaluate the safety of lbh589 given in combination with lenalidomide and dexamethasone in adult patients with multiple myeloma


Eligibility criteria:
inclusion criteria: * patients must have a diagnosis of active multiple myeloma * patients must have received at least one prior line of therapy and their disease has relapsed.. * patients must be suitable for treatment with lenalidomide \& dexamethasone. * adults ≥ 18 years old * ecog performance status ≤ 2 * life expectancy \> 12 weeks * patients must have acceptable neutrophil and platelet counts as well as adequate kidney and liver function. * able to sign informed consent and to comply with the protocol exclusion criteria: * primary refractory mm * peripheral neuropathy ≥ ctcae grade 2 * impaired cardiac function or clinically significant cardiac diseases * impairment of gi function or gi disease that may significantly alter the absorption of lbh589 * patients with diarrhea \> ctcae grade 1 * patients using medications that have a relative risk of prolonging the qt interval * concomitant use of cyp3a4 inhibitors * patients with a history of deep vein thrombosis or thromboembolism within \< 6 months prior to starting study treatment * patients who have undergone major surgery ≤ 4 weeks prior to starting study drug or who have not recovered from side effects of such therapy * women who are pregnant or breast feeding or women of childbearing potential (wocbp) not using 2 reliable forms of birth control * male patients whose sexual partners are wocbp and who are unable to use a latex condom during sexual contact (even if they have undergone a vasectomy) * patients with any significant history of non-compliance to medical regimens or unwilling or unable to comply with the instructions given to him/her by the study staff. other protocol defined inclusion/exclusion criteria may apply

Keywords: myeloma multiple myeloma lenalidomide revlimid dexamethasone lbh589 combination
a retrospective study: evaluation of the efficacy of immunotherapy with rare mutations in non-small cell lung cancer

Conditions: Non Small Cell Lung Cancer

Summary: in advanced non-small cell lung cancer, there will be some rare mutations, such as alk, kras, etc. the efficacy of these immunotherapies on these different rare mutations has not been reported. therefore, we conducted this retrospective clinical study to explore the efficacy of immunotherapy for different rare mutations.


Eligibility criteria:
inclusion criteria: * 1.diagnosed as advanced non-small cell lung cancer * 2.confirmed as kars, alk, erbb2, met, ret, braf mutation exclusion criteria: * the patient is diagnosed with small cell lung cancer
clinical feature and microbiology characteristics of empyema in children

Conditions: Empyema

Summary: the study is designed to clarify the clinical characteristics and reversion of children with empyema, observe the effect of streptococcus vaccine application on serotype of streptococcus, and to explore the susceptibility factors of streptococcus empyema in children in china.

empyema is a major complication of bacterial community-acquired pneumonia in children. and studies have shown that although the incidence of community-acquired pneumonia in children has decreased with the application of pneumococcal conjugate vaccine, the incidence of empyema has increased, which raises the difficulty to disease treatment, and aggravates the financial burden on medical care. streptococcus is one of the most common pathogens causing children's empyema. in recent years, although more and more researches are reported on children's community- acquired pneumonia or pleural effusion, the clinical studies on streptococcus pneumoniae empyema are rarely mentioned. therefore, a multicenter observational study is needed to better understand the clinical characteristics and reversion of children with empyema, observe the effect of streptococcus vaccine application on serotype of streptococcus, and explore the susceptibility factors of streptococcus empyema in children in china. the study mentioned above will contribute to optimize the treatment strategy of streptococcus empyema for the future, and reduce the incidence of children's empyema. the trial will be completed in 3 years with 100 participants taken from hospitals in partnership with clinical research collaboration of national clinical research center for respiratory diseases, china.

Eligibility criteria:
inclusion criteria: * age between 1 day to 18 years * admitted with diagnosis of pneumonia combined with pleural effusion * the appearance of thoracic fluid or the results of fluid examination conforms to empyema with at least 1 of the following criteria: * thoracentesis is pus. * thoracic fluid smear observation show bacterial-positive. * thoracic fluid culture test show bacterial-positive. * cell counting of thoracic fluid is more than 10x109/l. exclusion criteria: * subjects will be excluded if the thoracic fluids are caused by non-infectious factors, such as cardiovascular, connective tissue disease, tumor or trauma, etc.

Keywords: empyema streptococcus children
angiographic and intravascular ultrasound (ivus) follow-up after simultaneously implanted different drug-eluting stents in same individuals

Conditions: Drug Eluting Stent

Summary: the purpose of this study is to investigate if there is any different follow-up results by angiography and ivus between two different drug-eluting stents after implanted simultaneously in same individuals.


Eligibility criteria:
inclusion criteria: * these patients who received two kinds of drug-eluting stent (endeavor and excel) im simultaneously. exclusion criteria: * dual anti-platelet therapy less than 12 months.

Keywords: drug-eluting stent coronary restenosis coronary thrombosis
running retraining to minimize braking forces

Conditions: Athletic Injuries

Summary: the proposed research study aims to investigate whether a gait modification training program to decrease peak braking forces during the stance phase of running will result in a reduction in impact loading and the incidence of running-related injuries among recreational distance runners.


Eligibility criteria:
inclusion criteria: * ability to commit to a 15-week half-marathon training program * have been running for at least 3 months prior to study commencement * have participated in 2 or less half-marathons previously * ability to run on a treadmill unaided * ability to travel to testing facility for running analysis pre- and post-training program as well as for gait retraining sessions * ability to understand written and spoken english * meet the screening requirements (display less than -0.27bw mean peak braking force at baseline/screening assessment) exclusion criteria: * any lower extremity pathology in the previous 3 months or currently have pain in their lower back or lower extremities while running * have undergone hip, knee, or ankle joint surgery

Keywords: running biomechanics kinetics
long-term safety study of sublingual fentanyl tablets in cancer patients

Conditions: Pain; Cancer

Summary: the purpose of this study is to evaluate the long-term safety and effectiveness of en3267 in treating breakthrough pain episodes in opioid cancer patients who are using stable doses of opioid medication.

this was a phase 3 non-randomized, open-label, multicentre study designed to evaluate the long-term safety of en3267 in the treatment of btcp in opioid-tolerant cancer patients. the study was conducted in 2 parts: 1. a titration period during which patients had up to 2 weeks to determine a single, effective dose of study medication (en3267) for adequate treatment of btcp, and 2. a maintenance period of up to 12 months in which episodes of btcp were treated with study medication.

Eligibility criteria:
inclusion criteria: * males or females 17 years of age or older. * stable cancer-related pain. * are receiving a stable, fixed-schedule oral opioid regimen equivalent to 60 to 1000 mg of oral morphine per day or transdermal fentanyl therapy equivalent to 50 to 300 µg/h, and are on a stable dose of opioid medication for relief of breakthrough pain. * experiencing 1-4 episodes of breakthrough pain per day. * meet the criteria defined in the eastern cooperative oncology group (ecog) performance status for grade 0, 1, or 2. exclusion criteria: * have previously been exposed to en3267. * are pregnant or lactating. * have uncontrolled or rapidly escalating pain. * have any clinically significant condition that would, in the investigator's opinion, preclude participation in the study or compromise data collection. these conditions may include cardiopulmonary disease, and/or neurologic/psychologic conditions. * are scheduled to take maois (monoamine oxidase inhibitors) during the study. * are scheduled to receive anti-neoplastic therapy that, in the investigator's opinion, will influence assessment of breakthrough pain. * are scheduled to receive an investigational drug other than en3267 during the course of the study. * have hypersensitivity, allergy or contraindication to fentanyl. * have significant prior history of substance abuse or alcohol abuse. * would have difficulty complying with the protocol, as assessed by the investigator. * are unable to read, write, or comprehend the english language in order to complete diaries.

Keywords: en3267 breakthrough pain safety study fentanyl tablets
pulmonary rehabilitation and quality of life

Conditions: Coronary Artery Bypass Graft

Summary: in this study, the effect of a pulmonary rehabilitation (pr) program on the respiratory function and quality of life of patients undergoing cabg surgery with an open-heart technique is examined. a randomized controlled experimental design was used. the study was conducted with two groups: the intervention group (n=25) and control group (n=25). the control group received standard care after cabg. in contrast, the experimental group, in addition to standard care, participated in a pr program created by the researchers. after cabg, the respiratory functions of both groups (clinical care day 4) and quality of life (6th week) evaluated


Eligibility criteria:
inclusion criteria: * patients who were extubated in twenty-four hours, * aged 18 or above, * had undergone cabg surgery using the open-heart method and using a saphenous vein graft for cabg, * who were conscious, * without visual and hearing problems, * who spoke turkish, * had not orthopedic, psychiatric and neurological problems, * no co-morbid lung disease, * who had not been re-operated on, * had not postoperative cardiac dysfunction, * had not developed postoperative atrial fibrillation, * who were willing to participate in the study. exclusion criteria: * patients who were not extubated in twenty-four hours, * who were under 18 years of age, * had not undergone cabg surgery using the open-heart method and using a saphenous vein graft for cabg, * who were not conscious, * were visual and hearing problems, * who don't spoke turkish, * had orthopedic, psychiatric and neurological problems, * co-morbid lung disease, * who had been re-operated on, * had postoperative cardiac dysfunction, * had developed postoperative atrial fibrillation, * who were not willing to participate in the study.

Keywords: coronary artery bypass pulmonary rehabilitation quality of life respiratory functions
study of the jak inhibitor ruxolitinib administered orally to patients with primary myelofibrosis (pmf), post-polycythemia vera-myelofibrosis (ppv-mf) or post-essential thrombocythemia-myelofibrosis (pet-mf)

Conditions: Myelofibrosis

Summary: this is a phase ib, open-label, dose-finding study of the jak 1 and 2 inhibitor ruxolitinib in patients with myelofibrosis (mf). the study consists of two periods: the core study period, comprising the dose escalation stage and the safety extension phase up to week 24, then the extension study period beyond week 24 and up to 3 years, to further characterize the safety and efficacy of ruxolitinib in this patient population. the dose escalation phase will enroll successive cohorts of patients who receive increasing doses of ruxolitinib until the maximum safe starting dose (mssd) is determined. in the safety expansion phase, additional patients will be treated with ruxolitinib at the mssd defined during dose escalation. the primary objective is to establish the mssd of ruxolitinib in patients with mf and starting platelet counts \< 100 x 10 \^9/l


Eligibility criteria:
inclusion criteria: * require treatment for mf and classified at least as intermediate risk level 1 defined by the international working group. * platelet count \< 100x10 \^9/l at screening or at study day 1. exclusion criteria: * received platelet transfusion within 14 days prior to screening evaluations.